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2 articles from the last 30 days matching "aav9"

NewsRSS2 days ago

Test Your Knowledge About Life Expectancy in AAV

This article invites people to take a quiz about life expectancy in AAV (ANCA-associated vasculitis), a rare disease that affects blood vessels. After completing the quiz, you can see how your answers compare to other people who took it. The quiz is a way to learn more about what to expect with this condition.

WHY IT MATTERSUnderstanding life expectancy and prognosis in AAV can help patients and caregivers make informed decisions about treatment options and long-term planning.
ℹ Good to knowANCA-associated vasculitisRead →
Clinical trialCLINICALTRIALSApr 17

Trial Now Recruiting: A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial) (NCT04998396)

Researchers are testing a new gene therapy called BBP-812 to treat Canavan disease, a rare brain disorder that affects children. The therapy uses a modified virus to deliver a healthy copy of a gene that's missing or broken in people with this disease. This early-stage trial will check if the treatment is safe and whether it helps patients.

WHY IT MATTERSThis trial is now actively recruiting children with Canavan disease — if your child has been diagnosed, you may be eligible to participate in one of the first human tests of this gene therapy approach.
→ You can act on thisCanavan diseaseRead →

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